Ziftomenib with venetoclax and azacitidine in relapsed/refractory NPM1-mutated acute myeloid leukemia.
Eunice S Wang, Harry P Erba, Amer M Zeidan...
https://pubmed.ncbi.nlm.nih.gov/42227701/Actively Recruiting
Led by Kura Oncology, Inc. · Updated on 2026-03-13
420
Participants Needed
44
Research Sites
N/A
Total Duration
Researchers are studying ziftomenib, an investigational oral drug, in combination with standard treatments for patients with acute myeloid leukemia AML who have specific genetic mutations such as NPM1 or KMT2A rearrangements. This Phase 1 trial aims to assess the safety, tolerability, and early signs of effectiveness of ziftomenib when added to existing AML therapies in both newly diagnosed and relapsedrefractory patients. The study includes several treatment groups where ziftomenib is combined with drugs like venetoclax, azacitidine, daunorubicin, cytarabine, and quizartinib. Patients receive these medications through oral, intravenous, or subcutaneous routes depending on the combination. Different arms focus on distinct patient groups based on genetic markers and prior treatment history. The study investigates dose levels and combinations including intensive chemotherapy regimens and targeted therapies over multiple treatment cycles. Participants will be monitored closely from the first day of treatment through up to 36 months, with regular assessments of side effects, blood tests, and response to therapy including remission rates. Researchers will track dose-limiting toxicities and adverse events, as well as disease outcomes like complete remission and measurable residual disease. Safety and effectiveness data will be collected through a series of treatment cycles, with follow-up continuing for up to three years to evaluate long-term impact and survival outcomes.
CONDITIONS
A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML
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Kura Oncology, Inc.
Lead Sponsor
This table compares participation in this trial with other options available for this condition. The information is drawn from published research and the trial’s study details and is provided for educational purposes only.
| Factors | Ziftomenib + backbone (Ven/Aza, Ven, 7+3, or 7+3 + Quizartinib) | Standard-of‑care backbone regimens |
|---|---|---|
| What it is | An experimental menin‑inhibitor (ziftomenib) given with backbone regimens including venetoclax/azacitidine, venetoclax alone, 7+3 chemotherapy, or 7+3 plus quizartinib across cohorts | Established backbone regimens such as intensive chemotherapy (7+3) or venetoclax plus azacitidine used for patients with NPM1‑mutated or KMT2A‑rearranged AML depending on fitness |
| Role in trial | Single‑group investigational intervention across multiple cohorts | Contextual comparator reflecting regimens patients might otherwise receive |
| Primary use | Evaluated for safety, tolerability, and early efficacy in AML with NPM1 or KMT2A alterations | Used as frontline or relapsed backbone treatment based on patient fitness and molecular subtype |
| How it’s given | Oral ziftomenib added to oral venetoclax/azacitidine (oral/subcutaneous or IV), or to intravenous 7+3 with or without oral quizartinib | Venetoclax/azacitidine given orally/subcutaneously or IV; 7+3 delivered intravenously |
| Evidence available | Phase Ia/b data show high response rates: e.g., 94 % CRR with MRD negativity in newly diagnosed NPM1‑mutated AML; ORR ~65 % in R/R setting | Intensive chemotherapy yields CR rates up to ~85 % in fit patients; venetoclax/azacitidine defines care for older/unfit individuals |
| Important risks | Grade ≥3 treatment‑related adverse events include febrile neutropenia, cytopenias; differentiation syndrome reported | Intensive chemotherapy and venetoclax/azacitidine carry risks of cytopenias, infection, and standard chemotherapy toxicities |
| Important limitations | Single‑group, early‑phase study without randomized comparison; long‑term outcomes and survival advantage not yet known | Standard regimens can have substantial toxicity and may be poorly tolerated by older or frail patients |
| Patient impact | High early response and MRD‑negative remission may indicate deep remissions, but impact on long‑term survival is not yet established | Established regimens can achieve durable remission in fit patients though carry considerable side effects |
Disclaimer: This comparison is created with the help of artificial intelligence(AI) and may contain errors or missing information. It is not medical advice and does not mean one option is better or safer than another. Clinical trials are still being studied, and joining a trial is your choice. Please consult your healthcare professional before making any decisions.
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Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 36 months or until relapse, new anti-cancer therapy, death, or treatment discontinuation
Participants receive treatment with Ziftomenib in combination with Venetoclax/Azacitidine, Venetoclax, 7+3 chemotherapy, or 7+3+Quizartinib according to their assigned group.
Visits occur throughout each 28-day treatment cycle with assessments during and after treatment cycles
Duration - Up to 1 year following the start of treatment
Participants are monitored for safety, remission status, and long-term outcomes after treatment ends.
Periodic follow-up visits as scheduled by the study team
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
13
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Eunice S Wang, Harry P Erba, Amer M Zeidan...
https://pubmed.ncbi.nlm.nih.gov/42227701/Kura Oncology, Inc.
Lead Sponsor